NCT01233440

Brief Summary

The primary objective of the study is to assess the safety of IV administration of rIX-FP. Safety will be evaluated by adverse events and laboratory changes over time. The secondary objective of the study is to evaluate the pharmacokinetics parameters, following a single intravenous dose of rIX-FP.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Oct 2010

Geographic Reach
6 countries

20 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2010

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

November 2, 2010

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 3, 2010

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2011

Completed
Last Updated

January 31, 2012

Status Verified

January 1, 2012

Enrollment Period

9 months

First QC Date

November 2, 2010

Last Update Submit

January 26, 2012

Conditions

Hemophilia B

Outcome Measures

Primary Outcomes (4)

  • Frequency of adverse events (AEs)

    up to 14 days after drug administration

  • Frequency of serious adverse events (SAEs)

    up to 28 days after drug administration

  • Occurrence of inhibitor against FIX

    up to 28 days after drug administration

  • Occurrence of antibodies against rIX-FP

    up to 28 days after drug administration

Secondary Outcomes (5)

  • AUC to the last sample with quantifiable drug concentration (AUC0-t)

    From time of dosing up to 7 days after the dose

  • AUC extrapolated to infinity (AUCt-∞)

    From time of dosing up to 7 days after the dose

  • Half-life (t1/2)

    From time of dosing up to 7 days after the dose

  • Incremental recovery (IU/mL/IU/kg)

    From time of dosing up to 7 days after the dose

  • Clearance

    From time of dosing up to 7 days after the dose

Study Arms (3)

Cohort 1

EXPERIMENTAL

25 IU/kg dose

Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein

Cohort 2

EXPERIMENTAL

50 IU/kg dose

Biological: Recombinant Coagulation Factor IX Albumin Fusion ProteinBiological: Plasma derived FIX [pdFIX]

Cohort 3

EXPERIMENTAL

75 IU/kg dose

Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein

Interventions

Recombinant Coagulation Factor IX Albumin Fusion ProteinBIOLOGICAL

Single dose of 25, 50 or 75 IU/kg of rIX-FP, given as intravenous infusion

Cohort 1Cohort 2Cohort 3
Plasma derived FIX [pdFIX]BIOLOGICAL

Single dose of 50 IU/kg of reference product, given as intravenous infusion

Cohort 2

Eligibility Criteria

Age12 Years - 65 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Male, 12 - 65 years, with body weight ≥ 30 kg and ≤ 120 kg
  • Documented severe Hemophilia B (FIX activity of ≤ 2%) or tested by the central laboratory at screening
  • Subjects who have received FIX products for \> 150 exposure days (EDs) (estimated)
  • No confirmed prior history of FIX inhibitor (history of positive FIX inhibitor defined as two consecutive positive tests - a confirmatory test on a second, separately drawn sample shortly after the previous positive test) and confirmed no detectable FIX inhibitors (negative FIX inhibitor defined as \< 0.6 Bethesda Units \[BU\] by the central laboratory at screening
  • Subjects can be treated on-demand or under prophylactic therapy
  • Signed Informed Consent/Assent

You may not qualify if:

  • Known hypersensitivity (allergic reaction or anaphylaxis) to any FIX product or hamster protein
  • Any known congenital or acquired coagulation disorder other than congenital FIX deficiency
  • Platelet count \< 100,000/µL
  • Immunocompromised (CD4 count \< 200/mm3), (HIV positive subjects may participate in the study and protease inhibitors and antiviral therapy are permitted, at the discretion of the Investigator)
  • Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment
  • Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) concentration \> 5 times (x) the upper limit of normal (ULN)
  • Serum creatinine \> 2 x ULN
  • Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction and arterial embolus within 3 months prior to enrollment
  • Use of an Investigational Medicinal Product (IMP) within 30 days prior to the first rIX-FP administration
  • Experienced life-threatening bleeding episode or had major surgery or an orthopedic surgical procedure during the 3 months prior to study entry
  • Subject currently on a dose and/or regimen of FIX that would preclude participation in the study due to possible increased risk of bleeding because of the requirement to withhold treatment during the PK sampling period
  • Suspected inability (e.g., language problem or mental condition) or unwillingness to comply with study procedures or history of noncompliance

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Study site

Vienna, Austria

Location

Study site

Le Kremlin-Bicêtre, France

Location

Study Site

Lyon, France

Location

Study site

Nantes, France

Location

Study site

Paris, France

Location

Study Site

Berlin, Germany

Location

Study Site

Hamburg, Germany

Location

Study Site

Hanover, Germany

Location

Study site

Münster, Germany

Location

Study Site

Tel Litwinsky, Israel

Location

Study Site

Catania, Italy

Location

Study Site

Florence, Italy

Location

Study Site

Genova, Italy

Location

Study site

Milan, Italy

Location

Study Site

Napoli, Italy

Location

Study Site

Parma, Italy

Location

Study Site

Vicenza, Italy

Location

Study Site

A Coruña, Spain

Location

Study Site

Barcelona, Spain

Location

Study Site

Madrid, Spain

Location

Related Publications (1)

  • Santagostino E, Negrier C, Klamroth R, Tiede A, Pabinger-Fasching I, Voigt C, Jacobs I, Morfini M. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood. 2012 Sep 20;120(12):2405-11. doi: 10.1182/blood-2012-05-429688. Epub 2012 Aug 2.

    PMID: 22859609DERIVED

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Study Officials

  • Iris Jacobs, MD

    CSL Behring

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 2, 2010

First Posted

November 3, 2010

Study Start

October 1, 2010

Primary Completion

July 1, 2011

Study Completion

July 1, 2011

Last Updated

January 31, 2012

Record last verified: 2012-01

Locations

ES(3)AU(1)DE(4)IT(7)IL(1)FR(4)